Deep Brain Stimulation Surgery for Focal Hand Dystonia

NCT02911103 | Active Not Recruiting Phase 1 FDA Drug FDA Device

• 2 other identifiers: 16016616-N-0166
• Study Type: interventional
• Target: 5
• Locations: 1 country
• Sites: 1

Brief Summary

Researchers want to test a procedure called deep brain simulation (DBS) to treat focal hand dystonia (FHD). A device called a neurostimulator is placed in the chest. It is attached to wires placed in brain areas that affect movement. Stimulating these areas can help block nerve signals that cause abnormal movements. Objectives: To test DBS as treatment for FHD. To learn about brain and nerve cell function in people with dystonia. Eligibility: People ages 18 and older with severe FHD who have tried botulinum toxin treatment at least twice Design: Participation lasts 5 years. Participants will be screened with: Medical history Physical exam Videotape of their dystonia Blood, urine, and heart tests Brain MRI scan Chest X-ray Neuropsychological tests: answering questions, doing simple actions, and taking memory and thinking tests. Hand movement tests Participants will have surgery: A frame fixes their head to the operating table. A small hole is made in the skull. Wires are inserted to record brain activity and stimulate the brain while they do simple tasks. The wires are removed and the DBS electrode is inserted into the hole. The neurostimulator is placed under the skin of the chest, with wires running to the electrode in the brain. They will have CT and MRI scans during surgery. Participants will recover in the hospital for about 1 week. The neurostimulator will be turned on 1 4 weeks after discharge. Participants will have regular visits until the study ends. Visits include: Checking symptoms and side effects MRI Movement, thinking, and memory tests If the neurostimulator s battery runs out, participants will have surgery to replace it.

Conditions

Dystonia; Focal Dystonia; Musician's Dystonia

Keywords

Electrophysiology; Neurosurgery; Dystonia; Deep Brain Stimulation

Outcome Measures

Primary Outcomes (1)

• To confirm the safety of VOA/VOP thalamic DBS in FHD

  Number and severity of adverse events in the 5-year follow-up period.

  5 years

Secondary Outcomes (6)

• Neuropsychological Evaluation

  5 years

• To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in clinical improvement of severe intractable FHD

  5 years

• To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improved patient reported outcomes

  5 years

• To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improvement of severe intractable FHD in subjects with musician s dystonia

  5 years

• To determine if thalamic DBS targeting the VOA/VOP nuclear complex results in improvement of severe intractable FHD in subjects with Writer s cramp

  5 years

Study Arms (1)

Active

EXPERIMENTAL

single arm study

Procedure: DBS surgery; Device: Medtronic Activa Rechargeable System

Interventions

DBS surgery PROCEDURE

Unilateral thalamic DBS therapy

Active

Medtronic Activa Rechargeable System DEVICE

Used for diagnostic purposes.

Active

Eligibility Criteria

• Age: 22 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• FHD diagnosed by a neurologist
• BFM upper extremity subscore of 3 or more (score range 0 to 4) or ADDS difficulty of performing score equal to or more than 3.
• Patients must score 3 or below on the Tubiana and Chamagne scale or inability to perform at the concert level or patients must score a rating of "severe" on at least one item, impacting the patient s employment or essential activities on the Writer s Cramp Impairment scale.
• Symptoms causing significant impairment in quality of life and work or daily activities by patient self-assessment and SF12 scale, as reported by subjects. The subjects should be severely impaired in at least one of the following domains: handwriting, typing or playing an instrument. The impairment needs to have affected employment, manifested by substantive change in, or loss of, employment or career of choice.
• All patients must have failed or achieved inadequate benefit (continuing functional impairment) with at least two trials of botulinum toxin treatments.
• Women of childbearing age will have to agree to use contraception methods for the first 3 post operative months.

You may not qualify if:

• Subjects younger than 22 years old.
• MRI findings that would make participation and surgery unsafe (such as tumor or other space occupying lesion, stroke, hemorrhage or hematoma, edema, hydrocephalus, abscess) and MRI findings that would confound study outcomes (such as iron deposits in the basal ganglia, demyelinating disease, traumatic brain injury, structural or developmental abnormality, moderate or severe white matter hyperintensities).
• Subjects with prior brain surgery
• Pregnant or nursing women as safety of DBS has not been established in this group.
• Presence of a more widespread neurologic syndrome with the dystonia being part of it, and/or dystonia caused by an ongoing etiology, such as tardive dystonia associated with ongoing psychotropic treatment or an inherited neurodegenerative syndrome associated with intractable dystonia
• Intellectual disability as measured by the estimated General Ability Index (GAI) of the Wechsler Adult Intelligence Scale 4th Edition (WAIS-IV), which would render the participant not able to provide informed consent or to comply with the study procedures (estimated GAI less than 70)
• Dementia as evidenced by formal neuropsychological evaluation and Mattis Dementia Rating Scale-2 (DRS-2) score below 128.
• Depressed subjects as determined by the neuropsychology or psychiatry screen, including Beck Depression Inventory (BDI) and psychiatric evaluation. Subjects scoring above 20 on the BDI or exhibiting moderate or severe active depression will be excluded
• Subjects with uncontrolled co-existing medical conditions: uncontrolled systemic hypertension with values above 170/100; active heart disease needing immediate intervention; active respiratory disease needing immediate intervention; uncorrected coagulation abnormalities; any condition that would render the patient unable to safely cooperate with the surgery and study tests as judged by the screening physician
• Presence of a contraindication to undergo a brain MRI
• Metal in the body which would make having an MRI scan unsafe, such as pacemakers, medication pumps, aneurysm clips, metallic prostheses (including metal pins and rods, heart valves or cochlear implants), shrapnel fragments, permanent eye liner or small metal fragments in the eye that welders and other metal workers may have;
• Uncomfortable in small closed spaces (have claustrophobia) such that the participant will be uncomfortable in the MRI machine;
• Unable to lay comfortably flat on their back for up to one hour in the MRI scanner;
• Subjects who require diathermy
• Subjects who require post-op MRIs with full body coil

Sponsors & Collaborators

• National Institute of Neurological Disorders and Stroke (NINDS): lead

Study Sites (1)

National Institutes of Health Clinical Center

Bethesda, Maryland, 20892, United States

Location

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Dystonia; Dystonic Disorders; Dystonia, Focal, Task-Specific

Condition Hierarchy (Ancestors)

Dyskinesias; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Movement Disorders; Central Nervous System Diseases

Study Officials

• Debra J Ehrlich, M.D.

  National Institute of Neurological Disorders and Stroke (NINDS)

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 21, 2016
• First Posted: September 22, 2016
• Study Start: January 17, 2018
• Primary Completion (Estimated): February 2, 2029
• Study Completion (Estimated): February 2, 2029
• Last Updated: May 7, 2026

Record last verified: 2026-01-21

Locations

US (1)